Patients with myelodysplastic syndromes (MDS) who undergo hematopoietic cell transplantation (HCT) while still responding to hypomethylating agents (HMAs) have lower rates of post-HCT relapse, according to a study of 125 patients with MDS.
In this single-center investigation, median age was 61.4 years (range 30.4-77.2) and the median follow-up of survivors was 41.9 months (range, 2.7-98.5).
As shown in the table below, patients did best when they underwent HCT while still responding to HMAs (azacitidine, decitabine).
| HMA failures | HMA responders | p-value | |
| 3-year relapse | 56.6% | 34.2% | <0.01 |
| 3-year RFS | 23.8% | 42.0% | <0.01 |
The risk of non-relapse mortality was similar for both groups.
Clinical studies show that the prognosis of patients with MDS who fail HMA therapy is poor, and 40% to 60% of patients do not achieve clinically relevant responses to HMA therapy. HMA responses are typically of limited duration and the majority of patients show disease progression within 2 years.
This study supports that candidates for HCT should be referred for transplant consultation early in their treatment course, rather than waiting for HMA failure, as patients responding to HMAs at the time of HCT experience a significantly lower post-transplant incidence of relapse. In an accompanying editorial, Dr. Alison Loren notes that "fit patients with high-risk disease who can tolerate myeloablative conditioning probably will be best served with a direct-to-HCT approach."
Festuccia M, et al. Biol Blood Marrow Transplant